Publications
Listed below are citations for each publication. If you would like a copy of any of the publications,
please contact the GTRP Clinical Coordinating Center (gtrpccc@s-3.com).
2016
Vascular Regeneration in Ischemic Hindlimb by Adeno-Associated Virus Expressing Conditionally
Silenced Vascular Endothelial Growth Factor. Boden J, Lassance-Soares RM, Wang H, Wei Y, Spiga MG,
Adi J, Layman H, Yu H, Vazquez-Padron RI, Andreopoulos F, Webster KA. Journal of American Heart
Association. 2016 May 26;5(6).
Cardiac Stim1 Silencing Impairs Adaptive Hypertrophy and Promotes Heart Failure Through
Inactivation of mTORC2/Akt Signaling. Benard L, Gyun J, Cacheux M, Lee A, Nonnenmacher M, Matasic
DS, Kohlbrenner E, Kho C, Pavoine C, Hajjar RJ, Hulot JB. Circulation. 2016;133:1458-1471.
Intracoronary Gene Transfer of Adenylyl Cyclase 6 in Patients with Heart Failure: A Randomized
Clinical Trial. Hammond HK, Penny WF, Traverse JH, Henry TD, Watkins MW, Yancy CW, Swels RN, Adler
ED, Patel AN, Murray DR, Ross RS, Bhargava V, Maisel A, Barnard DD, Lai NC, Dalton ND, Lee ML,
Narayan SM, Blanchard DG, Gao MH. JAMA Cardiology. Online March 30, 2016.
Megakaryocyte- and megakaryocyte precursor-related gene therapies. Wilcox DA. Blood March 10
2016;127(10): 1260-1268
Safety and biodistribution assessment of sc-rAAV2.5IL-1Ra administered via intra-articular injection in
a mono-iodoacetate-induced osteoarthritis rat model. Wang G, Evans CH, Benson JM, Hutt JA,
Seagrave JC, Wilder JA, Grieger JC, Samulski RA, Terse PS. Molecular Therapy – Methods & Clinical
Development 2016 Jan 13;3:15052
2015
TrkB gene therapy by adeno-associated virus enhances recovery after cervical spinal cord injury.
Martinez-Galvez G, Zambrano JM, Diaz Soto JC, Zhan WZ, Gransee HM, Sieck GC, Mantilla CB.
Experimental Neurology 2015 Dec 1; 276:31-40
Safe and bodywide muscle transduction in young adult Duchenne muscular dystrophy dogs with
adeno-associated virus. Yue Y, Pan X, Hakim CH, Kodippili K, Zhang K, Shin JH, Yang HT, McDonald T,
Duan D. Human Molecular Genetics 2015 Oct 15;24(20):5880-90
Evaluation of Readministration of a Recombinant Adeno-Associated Virus Vector Expressing Acid
Alpha-Glucosidase in Pompe Disease: Preclinical to Clinical Planning. Corti M, Cleaver B, Clément N,
Conlon TJ, Faris KJ, Wang G, Benson J, Tarantal AF, Fuller D, Herzog RW, Byrne BJ. Human Gene Therapy
Clinical Development 2015 Sep;26(3): 185-93
1
Publications Cont’d
Listed below are citations for each publication. If you would like a copy of any of the publications,
please contact the GTRP Clinical Coordinating Center (gtrpccc@s-3.com)
2015 cont’d
Single and Compound Knock-outs of MicroRNA (miRNA)-155 and Its Angiogenic Gene Target CCN1in
Mice Alter Vascular and Neovascular Growth in the Retina via Resident Microglia. Yan L, Lee S, Lazzaro
DR, Aranda J, Grant MB, Chaqour B. Journal of Biological Chemistry 2015 Sep 18;290(38):23264-81
Progress with Recombinant Adeno-Associated Virus Vectors for Gene Therapy of Alpha-1 Antitrypsin
Deficiency. Gruntman AM, Flotte TR. Human Gene Therapy Methods. 2015 Jun;26(3):77-81
2014
A needleless liquid jet injection delivery method for cardiac gene therapy: a comparative evaluation
versus standard routes of delivery reveals enhanced therapeutic retention and cardiac specific gene
expression. Fargnoli AS, Katz MG, Williams RD, Margulies KB, Bridges CR. Journal of Cardiovascular
Translational Research 2014 Nov;7(8): 756-67
The Role of SUMO-1 in Cardiac Oxidative Stress and Hypertrophy. Lee A, Jeong D, Mitsuyama S, Oh JG,
Liang L, Ikeda Y, Sadoshima J, Hajjar R, Kho C. Antioxidants & Redox Signaling 2014 Nov 10;21(14): 19862001
Developmental stage determines efficiency of gene transfer to muscle satellite cells by in utero
delivery of adeno-associated virus vector serotype 2/9. Stitelman D, Brazelton T, Bora A, Traas J,
Joyeux L, Merianos D, Limberis MP, Davey MG, Flake AW. Molecular Therapy – Methods & Clinical
Development (2014) 1, 14040
Safety and efficacy of high-dose adeno-associated virus 9 encoding sarcoplasmic reticulum Ca (2+)
adenosine triphosphatase delivered by molecular cardiac surgery with recirculating delivery in ovine
ischemic cardiomyopathy. Katz MG, Fargnoli AS, Williams RD, Steuerwald NM, Isidro A, Ivanina AV,
Sokolova IM, Bridges CR. Journal of Thoracic Cardiovascular Surgery. 2014 Sep;148(3):1056-1073
Phase I/II Trial of Diaphragm Delivery of Recombinant Adeno-Associated Virus Acid Alpha-Glucosidase
(rAAV1-CMV-GAA) Gene Vector in Patients with Pompe Disease. Byrne BJ, Smith B, Mah C, Lawson LA,
Islam S, Corti M, Martin D, Dobija N, Irwin MV, Conlon T, Cleaver B, Clement N, Collins SW. Human Gene
Therapy Clinical Development 25: 134-163 (September 2014)
B-cell depletion is protective against anti-AAV capsid immune response: a human subject case study.
Corti M, Elder ME, Falk DJ, Lawson L, Smith BK, Nayak S, Conlon TJ, Clement N, Erger K, Lavassani E,
Green MM, Doerfler PA, Herzog RW, Byrne BJ. Molecular Therapy – Methods and Clinical Development
(2014) 1, 14033
2
Publications Cont’d
Listed below are citations for each publication. If you would like a copy of any of the publications,
please contact the GTRP Clinical Coordinating Center (gtrpccc@s-3.com).
2014 cont’d
In Utero Lung Gene Transfer Using Adeno-Associated Viral and Lentiviral Vectors in Mice. Joyeux L,
Danzer E, Limberis MP, Zoltick PW, Radu A, Flake AW, Davey MG. Human Gene Therapy Methods
25:197-205 (June 2014)
Assessment of toxicity and biodistribution of recombinant AAV8 vector-mediated immunomodulatory
gene therapy in mice with Pompe disease. Wang G, Young SP, Bali D, Hutt J, Li S, Benson J, Koeberl DD.
Molecular Therapy – Method & Clinical Development. 2014 June 11: 1-10
Attenuation of Monocrotaline-Induced Pulmonary Hypertension by Luminal Adeno-Associated Virus
Serotype 9 Gene Transfer of Prostacyclin Synthase. Gubrij IB, Martin SR, Pangle AK, Kurten R, Johnson
LG. Human Gene Therapy. 2014.Jun25(6):498-505
Targeted Delivery of TrkB Receptor to Phrenic Motorneurons Enhances Functional Recovery of
Rythmic Phrenic Activity After Cervical Spinal Hemisection. Gransee HM, Zhan WZ, Sieck GC, Mantilla
CB. PLoS One. 2014 May 28;8(5):1-10
Inhibition of miR-25 improves cardiac contractility in the failing heart. Wahlquist C, Jeong D, RojasMuñoz A, Kho C, Lee A, Mitsuyama S, van Mil A, Park WJ, Sluijter JP, Doevendans PA, Hajjar RJ, Mercola
M. Nature. 2014 Apr 24;508(7497):531-5
Preclinical Demonstration of Lentiviral Vector-mediated Correction of Immunological and Metabolic
Abnormalities in Models of Adenosine Deaminase Deficiency. Carbonaro DA, Zhang L, Jin X, MontielEquihua C, Geiger S, Carmo M, Cooper A, Fairbanks L, Kaufman ML, Sebire NJ, Hollis RP, Blundell MP,
Senadheera S, Fu P, Sahaghian A, Chan RY, Wang X, Cornetta K, Thrasher AJ, Kohn DB, Gaspar HB.
Molecular Therapy. 2014 Mar; 22(3):607-22
What is suppression of anti-adeno-associated virus capsid T-cells achieving? Flotte TR, Mueller C.
Human Gene Therapy. 2014 Mar;25(3):178-9
2013
Intrapleural Administration of an AAVrh.10 Vector Coding for Human α1-Antitrypsin for the
Treatment of α1-Antitrypsin Deficiency. Chiuchiolo MJ, Kaminsky SM, Sondhi D, Hackett NR, Rosenberg
JB, Frenk EZ, Hwang Y, Van de Graaf BG, Hutt JA, Wang G, Benson J, Crystal RG. Human Gene Therapy
Clinical Development. 2013 Dec;24(4):161-73
3
Publications Cont’d
Listed below are citations for each publication. If you would like a copy of any of the publications,
please contact the GTRP Clinical Coordinating Center (gtrpccc@s-3.com)
2013 cont’d
Human Treg responses allow sustained recombinant adeno-associated virus-mediated transgene
expression. Mueller C, Chulay JD, Trapnell BC, Humphries M, Carey B, Sandhaus RA, McElvaney NG,
Messina L, Tang Q, Rouhani FN, Campbel-Thompson M, Fu AD, Yachnis A, Knop DR, Ye GJ, Brantly M,
Calcedo R, Somanathan S, Richman LP, Vonderheidi RH, Hulme MA, Brusko TM, Wilson JM, Flotte TR.
Journal of Clinical Investigation. 2013 Dec:123(12):5310-8
SUMO-1 gene transfer improves cardiac function in a large-animal model of heart failure. Tilemann L,
Lee A, Ishikawa K, Aguero J, Rapti K, Santos-Gallego C, Kohlbrenner E, Fish KM, Kho C,Hajjar RJ. Science
Translational Medicine. 2013 Nov 3;5(211):211ra159
Retinol-binding protein 4 and its membrane receptor STRA6 control adipogenesis by regulating
cellular retinoid homeostasis and retinoic acid receptor α activity. Muenzner M, Tuvia N,
Deutschmann C, Witte N, Tolkachov A, Valai A, Henze A, Sander LE, Raila J, Schupp M. Molecular and
Cellular Biology. 2013 Oct;33(20):4068-82
Adeno-associated virus serotype 9 efficiently targets ischemic skeletal muscle following systemic
delivery. Katwal AB, Konkalmattt PR, Piras BA, Hazarika S, Li SS, Lye R. John, Sanders JM, Ferrante EA,
Yan Z, Annex BH, French BA. Gene Therapy. 2013 Sep;20(9):930-8
Cardiac surgical delivery of the sarcoplasmic reticulum calcium ATPase rescues myocytes in ischemic
heart failure. Fargnoli AS, Katz MG, Yarnall C, Isidro A, Petrov M, Steuerwald N, Ghosh S, Richardville
KC, Hillesheim R, Williams RD, Kohlbrenner E, Stedman HH, Hajjar RJ, Bridges CR. Annals of Thoracic
Surgery. 2013 Aug;96(2):586-95.
Phase I/II Trial of Adeno-Associated Virus-Mediated Alpha-Glucosidase Gene Therapy to the
Diaphragm for Chronic Respiratory Failure in Pompe Disease: Initial Safety and Ventilatory Outcomes.
Smith BK, Collins SW, Conlon TJ, Mah CS, Lawson LA, Martin AD, Fuller DD, Cleaver BD, Clément N,
Phillips D, Islam S, Dobjia N, Byrne BJ. Human Gene Therapy. 2013 Jun;24(6):630-40
Microdystrophin ameliorates muscular dystrophy in the canine model of Duchenne Muscular
Dystrophy. Shin JH, Pan X, Hakim CH, Yang HT, Yue Y, Zhang K, Terjung RL, Duan D. Molecular Therapy.
2013 Apr;21(4):750-57
Molecular mechanisms responsible for the differential effects of ApoE3 and ApoE4 on plasma
lipoprotein – cholesterol levels. Li H, Dhanasekaran P, Alexander ET, Rader DJ, Phillips MC, Lund-Katz S.
Arteriosclerosis, Thrombosis, and Vascular Biology. 2013 Apr;33(4):687-93
4
Publications Cont’d
Listed below are citations for each publication. If you would like a copy of any of the publications,
please contact the GTRP Clinical Coordinating Center (gtrpccc@s-3.com)
2012
Lentiviral Vector Gene Transfer to Porcine Airways. Sinn PL, Cooney AL, Oakland M, Dylla DE, Wallen
TJ, Pezzulo AA, Chang EH, McCray PB Jr. Molecular Therapy-Nucleic Acids. 2012 Nov 27
Connective tissues growth factor regulates retinal neovascularization through p53 protein-dependent
transactivation of the Matrix Metalloproteinase (MMP)-2. Chintala H, Liu H, Parmar R, Kamalska M,
Kim YJ, Lovett D, Grant MB, Chaqour B. Journal of Biological Chemistry. 2012 Nov 23;287(48):40570-85
Hepatic sortilin regulates both apolipoprotein B secretion and LDL catabolism. Strong A, Ding Q,
Edmondson AC, Millar JS, Sachs KV, Li X, Kumaravel A, Wang MY, Ding A, Guo L, Alexander ET, Nguyen D,
Lund-Katz S, Phillips MC, Morales CR, Tall AR, Kathiresan S, Fisher EA, Musunuru K, Rader DJ. Journal of
Clinical Investigation. 2012 August 1;122(8); 2807-2816
Successful transfection of genes using AAV-2/9 vector in swine coronary and peripheral arteries.
Pankajakshan D, Makinde TO, Gaurav R, Del Core M, Hatzoudis G, Pipinos I, Agrawal DK. Journal of
Surgical Research. 2012 Jun 1;175(1):169-175
Characterization of an HIV-targeted transcriptional gene-silencing RNA in primary cells. Turner AMW,
Ackley AM, Matrone MA, Morris KV. Human Gene Therapy. 2012 May;23:473-483
An AAV2/5 vector enhances safety of gene transfer to the mouse salivary gland. Geguchadze RN,
Machen L, Zourelias L, Gallo PH, Passineau MJ. Journal of Dental Research. 2012 Apr;91(4):382-6
Humoral immunity to AAV-6, 8, and 9 in normal and dystrophic dogs. Shin JH, Yue Y, Smith B, Duan D.
Human Gene Therapy. 2012 March;23:287-294
Analysis of particle content of recombinant adeno-associated virus serotype 8 vectors by ionexchange chromatography. Lock M, Alvira MR, Wilson JM. Human Gene Therapy Methods. 2012
Feb;23(1):56-64
A simplified immune suppression scheme leads to persistent micro-dystrophin expression in
Duchenne Muscular Dystrophy dogs. Shin JH, Yue Y, Srivastava A, Smith B, Duan D. Human Gene
Therapy. 2012 Feb;23:202-209
The effects of apolipoprotein F deficiency on high density lipoprotein cholesterol metabolism in mice.
Lagor WR, Fields DW, Khetarpal SA, Kumaravel A, Lin W, Weintraub N, Wu K, Hamm-Alvarez SF, DrazulSchrader D, de la Llera-Moya M, Rothblat GH, Rader DJ. PLoS ONE. 2012 Feb;7(2):e31616
5
Publications Cont’d
Listed below are citations for each publication. If you would like a copy of any of the publications,
please contact the GTRP Clinical Coordinating Center (gtrpccc@s-3.com)
2012 cont’d
Developing novel lentiviral vectors into clinical products. Leath A and Cornetta K. Methods in
Enzymology. 2012;507:89-108
2011
Phase 2 clinical trial of a recombinant adeno-associated viral vector expressing α1-antitrypsin: interim
results. Flotte TR, Trapnell BC, Humphries M, Carey B, Calcedo R, Rouhani F, Campbell-Thompson M,
Yachnis AT, Sandhaus RA, McElvaney NG, Mueller C, Messina LM, Wilson JM, Brantly M, Knop DR, Ye GJ,
Chulay JD. Human Gene Therapy. 2011 Oct;22(10):1239-47
Myocardial gene delivery using molecular cardiac surgery with recombinant adeno-associated virus
vectors in vivo. White JD, Thesier DM, Swain JBD, Katz MG, Tomasulo C, Henderson A, Wang L, Yarnall
C, Fargnoli A, Sumaroka M, Isidro A, Petrov M, Holt D, Nolen-Walston R, Koch WJ, Stedman HH,
Rabinowitz J, Bridges CR. Gene Therapy (2011) 18; 546-552
PEG-modulated column chromatography for purification of recombinant adeno-associated virus
serotype 9. Zhou J, Yang X, Wright JF, High KA, Couto L, Qu G. Journal of Virology Methods. 2011
Apr;173(1):99-107.
The matricellular protein cysteine-rich protein 61 (CCN1/Cyr61) enhances physiological adaptation of
retinal vessels and reduces pathological neovascularization associated with ischemic retinopathy.
Hasan A, Pokeza N, Shaw L, Lee HS, Lazzaro D, Chintala H, Rosenbaum D, Grant MB, Chaqour B. Journal
of Biological Chemistry. March 18, 2011;286(11):9542-9554
Preclinical evaluation of a recombinant adeno-associated virus vector expressing human alpha-1
antitrypsin made using a recombinant herpes simplex virus production method. Chulay JD, Ye GJ,
Thomas DL, Knop DR, Benson JM, Hutt JA, Wang G, Humphries M, Flotte TR. Human Gene Therapy. 2011
Feb;22:155-165
Vector characterization methods for quality control testing of recombinant adeno-associated viruses.
Wright JF and Zelenaia O. Methods in Molecular Biology. 2011;737:247-78
2010
Gene therapy during cardiac surgery: role of surgical technique to minimize collateral organ gene
expression. Katz MG, Swain JD, Fargnoli AS, Bridges CR. Interactive Cardiovascular and Thoracic
Surgery. 2010 Dec;11(6):727-31
6
Publications Cont’d
Listed below are citations for each publication. If you would like a copy of any of the publications,
please contact the GTRP Clinical Coordinating Center (gtrpccc@s-3.com)
2010 cont’d
Trib1 is a lipid- and myocardial infarction–associated gene that regulates hepatic lipogenesis and VLDL
production in mice. Burkhardt R, Toh SA, Lagor WR, Birkeland A, Levin M, Li X, Robblee M, Fedorov VD,
Yamamoto M, Satoh T, Akira S, Kathiresan S, Breslow JL, Rader DJ. The Journal of Clinical Investigation.
2010;120(12):4410–4414
Specific disruption of astrocytic Ca2+ signaling pathway in vivo by adeno-associated viral transduction.
Xie Y, Wang T, Sun GY, Ding S. Neuroscience. 2010 Nov 10;170(4):992-1003
Rapid, simple, and versatile manufacturing of recombinant Adeno-associated viral vectors at scale.
Lock M, Alvira M, Vandenberghe LH, Samanta A, Toelen J, Debyser Z, Wilson JM. Human Gene Therapy.
2010 Oct;21(10):1259-1271
Efficient serotype-dependent release of functional vector into the culture medium during Adenoassociated virus manufacturing. Vandenberghe LH, Xiao R, Lock M, Lin J, Korn M, Wilson JM. Human
Gene Therapy. 2010 Oct;21(10):1251-1257
From noncoding variant to phenotype via SORT1 at the 1p13 cholesterol locus. Musunuru K, Strong A,
Frank-Kamenetsky M, Lee NE, Ahfeldt T, Sachs KV, Li X, Li H, Kuperwasser N, Ruda VM, Pirruccello JJ,
Muchmore B, Prokunina-Olsson L, Hall JL, Schadt EE, Morales CR, Lund-Katz S, Phillips MC, Wong J,
Cantley W, Racie T, Ejebe KG, Orho-Melander M, Melander O, Koteliansky V, Fitzgerald K, Krauss RM,
Cowan CA, Kathiresan S, Rader DJ. Nature. 2010 Aug 5; 466(7307):714-9
2009
Synergistic effect of HIF-1alpha gene therapy and HIF-1-activated bone marrow-derived angiogenic
cells in a mouse model of limb ischemia. Rey S, Lee K, Wang CJ, Gupta K, Chen S, McMillan A, Bhise N,
Levchenko A, Semenza GL. Proceedings of the National Academy of Sciences USA. 2009 Dec
1;106(48):20399-404
Liver-specific deficiency of serine palmitoyltransferase (SPT) subunit 2 decreases plasma
sphingomyelin and increases Apolipoprotein E levels. Li Z, Li Y, Chakraborty M, Fan Y, Bui HH, Peake DA,
Kuo MS, Xiao X, Cao G, Jiang XC. Journal of Biological Chemistry. 2009 Sep 25;284(39):27010-9
Overexpression of Apolipoprotein F reduces HDL cholesterol levels in vivo. Lagor WR, Brown RJ, Toh S,
Millar JS, Fuki IV, de la Llera-Moya M, Yuen T, Rothblat G, Billheimer JT, Rader DJ. Arteriosclerosis,
Thrombosis, and Vascular Biology. 2009 Jan 29(1):40-6
7
Presentations
Listed below are citations for each presentation. If you would like a copy of any of the presentations,
please contact the GTRP Clinical Coordinating Center (gtrpccc@s-3.com).
2016
Gene Therapy Reduces Angioplasty induced Coronary Restenosis in a High Cholesterol Swine Model.
Agrawal DK et al. European Atherosclerosis Society. 2016 May 29 – June 1 (slides)
A Characterization of Transgene-Specific “Proteomes” in Lentiviral Vector Clinical Production Lots
Identifies Differences Between Vectors with High and Low Infectious Titers. Witting S, Reeves L,
Witzmann F. American Society of Cell and Gene Therapy Annual Meeting. 2016 May 4-7 (poster)
2015
Gene Therapy Using a Self-Inactivating Lentiviral Vector Improves Clinical and Laboratory
Manifestations of Wiskott-Aldrich Syndrome. Chu JI, Henderson LA, Armant M, Male F, Dansereau CH,
MacKinnon B, Burke CJ, Cavanaugh ME, London WB, Barlan IB, Ozen A, Baris S, Karakoc-Aydiner E,
Kiykim A, Hanson IC, Despotovic JM, Saitoh A, Takachi T, Imai K, King A, Arredondo S, Ritz J, Bushman FD,
Galy A, Daniele L, Williams DA, Pai SY. American Society of Hematology Annual Meeting. 2015
December 6 (slides)
Mesenchymal stem cells in the prevention of thrombosis and intimal hyperplasia. Agrawal DK et al.
Great Wall – International Congress of Cardiology. 2015 October 29 – November 1 (slides)
Gene Therapy to Prevent Restenosis Following Coronary Intervention. Agrawal DK et al. International
Congress of Cardiology. 2015 October 22-24 (slides)
Differentiation of Bone Marrow Stromal Cells Into Endothelial Cells and Stem Cell Therapy in Coronary
Artery Disease. Agrawal DK et al. International Academy of Cardiovascular Sciences – European
section. 2015 October 7-10 (slides)
AC6 Gene Transfer for Clinical CHF. Hammond HK. 2015 European Society of Cardiology Congress. 2015
September (slides)
Gene Therapy to Prevent Restenosis Following Intervention in CAD. Agrawal DK et al. 8th
International Congress on Cardiovascular Disease. 2015 August 13-15 (slides)
Gene Therapy to Prevent Restenosis Following Coronary Intervention. Agrawal DK et al. Invited
Presentation at the University of Missouri. 2015 May 12 (slides)
Stem Cell Therapy in Coronary Artery Disease. Agrawal DK et al. Invited Presentation at the
Regenerative Medicine Symposium. 2015 April 17 (slides)
8
Presentations Cont’d
Listed below are citations for each presentation. If you would like a copy of any of the presentations,
please contact the GTRP Clinical Coordinating Center (gtrpccc@s-3.com).
2015 Cont’d
Gene Therapy to Prevent Restenosis Following Coronary Intervention. Agrawal DK et al. Invited
Presentation at the University of Nebraska Medic al Center. 2015 January 26 (slides)
2014
An Innovative Needleless Liquid Jet Injection Delivery Method for Heart Failure Gene Therapy.
Fargnoli AS. 2014 Heart Failure Society Meeting. 2014 November (slides)
Safety and Efficacy of High Dose SERCA2a Gene Therapy Delivered by Molecular Cardiac Surgery with
Recirculating Delivery (MCARD). Bridges C. American Association of Thoracic Surgery Annual Meeting.
2014 April (slides)
Targeted Glyoxalase-1 gene transfer prevents cardiac dysfunction in diabetes. Bidasee K. Experimental
Biology Meeting. 2014 April (slides)
Targeted Glyoxalase-1 gene transfer to prevent cerebral vascular dysfunction and cognitive decline in
rates with type-1 diabetes. Bidasee K. Experimental Biology Meeting. 2014 April (poster)
2013
Gene therapy for α1-antitrypsin deficiency with AAV intrapleural administration. Crystal R. American
Society of Gene and Cell Therapy Annual Meeting. Salt Lake City, UT. 2013 May (slides)
2012
ApoE3 and ApoE4 polymorphism: Mechanistic basis for differential isoform effects on serum
cholesterol levels and lipoprotein profiles. Lund-Katz S. Recent Advances in Cardiovascular Disease
Assessment, XVI International Symposium on Atherosclerosis, Sydney, Australia. 2012 March (slides)
2011
Normalization of angiogenesis and vasculogenesis by CCN1: Implications in occular neovascular
diseases. Chaqour B. The International CCN Society: Targets 2011, Vancouver, BC, Canada. 2011
September 24-27 (slides)
Transductional and transcriptional targeting of AAV to the pulmonary vasculature. Passineau MJ,
Zourelias L, Machen L, Benza RL. American Thoracic Society 2011, Denver, Colorado. 2011 May 13-18
(poster)
9
Presentations Cont’d
Listed below are citations for each presentation. If you would like a copy of any of the presentations,
please contact the GTRP Clinical Coordinating Center (gtrpccc@s-3.com).
2011 Cont’d
Comparison of the structure-function relationships of human ApoE3 and ApoE4: From in vitro
structural studies to in vivo mouse studies. Lund-Katz S. American Heart Association Workshop on
HDL, Chicago, Illinois. 2011 April (slides)
2010
Adeno-associated virus 9 vector in the gene therapy of occlusive vascular diseases. Agrawal DK,
Pankajakshan D, Del Core M, Pipinos I, Hatzoudis G. European Atherosclerosis Society Congress 2010,
Hamburg, Germany. 2010 June 20-23 (poster)
The next step from GWAS and eQTL candidate gene discovery: An in vivo follow up of novel genes
identified through the Global Lipids Genetics Consortium. Stylianou IM, Fitzgerald K, Kathiresan S,
Rader DJ. 9th Annual Meeting of the Complex Trait Community, Chicago, Illinois. 2010 May 10 (slides)
Characterization of GALNT2, a novel GWAS locus associated with HDL-C. Edmondson AC, Khera A,
Khetarpal SA, Kathiresan S, Rader DJ. Arteriosclerosis, Thrombosis and Vascular Biology (ATVB) 2010
Scientific Sessions, San Francisco, California. 2010 April 8-10 (poster)
Sortilin is a novel modulator of low-density lipoprotein cholesterol. Strong A, Musunuru K, FrankKamenetsky M, Sachs KV, Li X, Li H, Lund-Katz S, Phillips M, Wong J, Cantley W, Koteliansky V, Fitzgerald
K, Krauss RM, Kathiresan S, Rader DJ. Arteriosclerosis, Thrombosis and Vascular Biology (ATVB) 2010
Scientific Sessions, San Francisco, California. 2010 April 8 (slides)
2009
AAV9-Wnt11 gene therapy improves cardiac recovery after myocardial infarction by modulating the
inflammatory response. Kobayashi K, Klyachko E, Thorne T, Kentaro J, Tongers J, Misener S, Losordo
DW. American Heart Association Meeting, Orlando, Florida. 2009 November 14-18 (slides)
Hemeoxygenase-1 gene modification enhances the regenerative efficacy of CD34+ cell-therapy after
myocardial infarction. Tongers J, Kentaro J, Roncalli JG, Renault MA, Kamide CE, Klyachko E, Kobayashi
K, Motlagh D, Losordo DW. American Heart Association Meeting, Orlando, Florida. 2009 November 1418 (slides)
Functional characterization of GALNT2, a novel GWAS locus associated with HDL-C. Edmondson A,
Khera A, Khetarpal S, Kathiresan S, Rader DJ. 59th Annual Meeting of the American Society of Human
Genetics, Honolulu, Hawaii. 2009 October 24 (slides)
10
Presentations Cont’d
Listed below are citations for each presentation. If you would like a copy of any of the presentations,
please contact the GTRP Clinical Coordinating Center (gtrpccc@s-3.com).
2009 cont’d
Functional characterization of GALNT2, a novel GWAS locus associated with HDL-C. Edmondson A,
Khera A, Khetarpal S, Kathiresan S, Rader D. Gordon Research Conference, Human Genetics and
Genomics, Biddeford, Maine. 2009 July 19-24 (poster)
11